Clinical Trials

Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Biological
  • Participants: 6
  • Start Date: March 12, 2021
Phase 1/2, Open-Label Clinical Study to Evaluate the Safety and Efficacy of Intrathecal TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis
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Clinical Trial
  • Status: Recruiting
  • Phase: Phase 2
  • Intervention Type: Drug
  • Participants: 39
  • Start Date: June 7, 2019
Effects of N-Acetyl-L-Leucine on GM2 Gangliosdisosis (Tay-Sachs and Sandhoff Disease): A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 3
  • Intervention Type: Drug
  • Participants: 30
  • Start Date: January 14, 2019
Survey of Miglustat Therapeutic Effects on Neurological and Systemic Symptoms of Infantile Type of Sandhoff and Taysachs Diseases
Clinical Trial
  • Status: Recruiting
  • Participants: 15
  • Start Date: April 2016
A Natural History of Late Onset Tay-Sachs Disease: MGH Site
Clinical Trial
  • Status: Recruiting
  • Intervention Type: Biological, Other
  • Participants: 1500
  • Start Date: January 11, 2012
Longitudinal Study of Neurodegenerative Disorders
Clinical Trial
  • Status: Recruiting
  • Participants: 52
  • Start Date: December 2010
A Natural History Study of the Gangliosidoses
Clinical Trial
  • Status: Recruiting
  • Participants: 200
  • Start Date: January 25, 2002
Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
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